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Vidaza (azacitidine)
Vidaza works by incorporating itself into DNA and interfering with the enzyme that adds methyl groups, which helps to restore normal gene function in cancer cells.
At a glance
| Generic name | azacitidine |
|---|---|
| Sponsor | Bristol-Myers Squibb |
| Drug class | Nucleoside Metabolic Inhibitor [EPC] |
| Target | DNA (cytosine-5)-methyltransferase 1 |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2004 |
Mechanism of action
VIDAZA is pyrimidine nucleoside analog of cytidine. VIDAZA is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of azacitidine cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to azacitidine.
Approved indications
- Refractory Anemia (RA)
- Refractory Anemia with Ringed Sideroblasts (RARS)
- Refractory Anemia with Excess Blasts (RAEB)
- Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
- Chronic Myelomonocytic Leukemia (CMMoL)
Common side effects
- anemia
- febrile neutropenia
- leukopenia
- neutropenia
- thrombocytopenia
- constipation
- diarrhea
- nausea
- vomiting
- pyrexia
- nasopharyngitis
- pneumonia
Key clinical trials
- Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission (PHASE3)
- Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML (PHASE1)
- The Safety and Tolerability of LBS-007 in Patients With Relapsed or Resistant Acute Leukaemias (PHASE1,PHASE2)
- BLAST MRD AML-2: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 2- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Azacitidine and Venetoclax as Frontline Therapy in Unfit Patients With Acute Myeloid Leukemia (PHASE2)
- Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia (PHASE2)
- Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial) (PHASE2)
- Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
| 8846628 | 2030-06-03 | Formulation |
| 12053482 | 2029-05-14 | Method of Use |
| 11571436 | 2029-05-14 | Formulation |
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Vidaza CI brief — competitive landscape report
- Vidaza updates RSS · CI watch RSS
- Bristol-Myers Squibb portfolio CI